Clinical Study of Eflornithine After Immunotherapy for High-risk Neuroblastoma（CSHEIN） (CSHEIN)

December 11, 2025 updated by: Shanghai Children's Hospital
Evaluate the impact of maintenance therapy with eflornithine on event-free survival and overall survival in high-risk neuroblastoma (NB) children after immunotherapy, and assess its safety.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Detailed Description

The investigators plan to recruit 20 participants and divide them into Group A and Group B based on their clinical status. The inclusion criteria for Group A are: newly diagnosed high-risk neuroblastoma (NB) patients who have completed the standard treatment plan (including immunotherapy) and whose disease has reached complete response (CR) or very good partial response (VGPR). The inclusion criteria for Group B are: recurrent or refractory NB patients who have completed any treatment for recurrent disease, or who have achieved disease stability after any salvage or intensification therapy for primary refractory disease, with at least a partial response (PR) as assessed by CT or MRI and negative bone marrow aspiration. There are no restrictions regarding gender or geographic region. Participants must be under 18 years of age. Eligible subjects will be enrolled into the study after receiving the first oral dose of eflornithine, with a total treatment course of 2 years. Follow-up will continue for up to 3 years after completion of eflornithine therapy.

Study Type

Observational

Enrollment (Estimated)

20

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
      • Hainan, China
        • Ruijin Hospital Hainan Branch, affiliated with Shanghai Jiao Tong University School of Medicine and Children's Hospital of Shanghai, affiliated with Shanghai Jiao Tong University School of Medicine

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Children with high-risk neuroblastoma

Description

Inclusion Criteria:

  1. According to the International Neuroblastoma Risk Group Classification, it has been histologically diagnosed as high-risk NB.
  2. Under the age of 18.
  3. The disease assessment status is PR (via CT or MRI) and the bone marrow smear is negative.
  4. If the residual mass is MIBG negative or MIBG positive and lacks FDG-PET affinity, it is considered evidence that the mass does not represent active disease. Subjects with stable residual tumor mass visible on CT/MRI will be included in the study.
  5. Qualified hematological parameters and organ function; Refer to the CI CTC 4.0 adverse reaction grading standard of grade 2 and below.
  6. Eflornithine needs to be activated within 120 days after the completion of previous treatment.

Exclusion Criteria:

  1. According to the International Neuroblastoma Risk Group Classification, it has been histologically diagnosed as non high risk NB.
  2. Prior to enrollment, the disease assessment status was either progressive disease (PD) or relapse (via CT or MRI).
  3. Positive bone marrow smear.
  4. Hematological parameters and organ function are not qualified, according to the CI CTC 4.0 adverse reaction grading standard of grade 3 or above.
  5. The guardian does not agree to participate.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The event free survival rate after the completion of the first DFMO
Time Frame: 5 years
The event free survival rate of high-risk NB patients who complete standard treatment and take oral eflornithine after immunotherapy.
5 years
overall survival rate after the completion of the first DFMO
Time Frame: 5 years
overall survival rate of high-risk NB patients who complete standard treatment and take oral eflornithine after immunotherapy.
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence of Treatment-Emergent Adverse Events
Time Frame: 5 years
This study will comprehensively evaluate the safety and tolerability of eflornithine. The primary assessments include the incidence and severity of all treatment-emergent adverse events (graded per NCI CTCAE v5.0)
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shanghai Children's Hospital

Collaborators

Ruijin Hospital

Investigators

  • Principal Investigator: Jingbo Shao, PhD，chief physician, Shanghai Children's Hospital
  • Principal Investigator: Wen Su, PhD，associate chief physician, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 8, 2025

Primary Completion (Estimated)

October 31, 2029

Study Completion (Estimated)

October 31, 2029

Study Registration Dates

First Submitted

February 8, 2025

First Submitted That Met QC Criteria

December 11, 2025

First Posted (Actual)

December 12, 2025

Study Record Updates

Last Update Posted (Actual)

December 12, 2025

Last Update Submitted That Met QC Criteria

December 11, 2025

Last Verified

December 1, 2025

More Information

Terms related to this study

Keywords

Other Study ID Numbers

  • ShanghaiChildrens

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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